Brexu-cel in R/R B-ALL with CNS involvement: Real-world outcomes

Results from a retrospective multicenter analysis of brexucabtagene autoleucel (brexu-cel) in patients with B-cell acute lymphoblastic leukemia (B-ALL) with central nervous system (CNS) involvement, using data from the Real-World Outcomes Collaborative for CAR T in Adult ALL (ROCCA) consortium, were recently published in Blood Advances by Muhsen et al.

Key data: Of 189 patients who received infusion with brexu-cel, 31 had active CNS disease at the pre-apheresis disease assessment and 158 had no reported CNS disease. In the CNS group, after brexu-cel (n = 24), 21 patients (87.5%) achieved CNS-1 (no identifiable CNS disease) and three patients continued to have active CNS disease. Comparable toxicity profiles were observed between CNS and non-CNS groups, with cytokine release syndrome (CRS) occurring in 74.2% vs 84.8% and immune effector cell-associated neurotoxicity syndrome (ICANS) in 64.5% vs 54.4%, respectively. No statistical differences were observed between progression-free survival (PFS) or overall survival (OS) in patients with or without CNS involvement. 

Key learning: Treatment with brexu-cel resulted in high rates of CNS disease remission in patients with relapsed/refractory (R/R) B-ALL with CNS involvement, with comparable efficacy and safety to that observed in patients without CNS involvement. These results support the inclusion of patients with CNS B-ALL in future chimeric antigen receptor (CAR) T-cell clinical trials, while highlighting the need for continued treatment optimization in this population.